Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene

Dolan Sondhi, Emma C. Scott, Alvin Chen, Neil R. Hackett, Andrew M.S. Wong, Agnieszka Kubiak, Hemanth R. Nelvagal, Yewande Pearse, Susan L. Cotman, Jonathan D. Cooper, Ronald G. Crystal

Research output: Contribution to journalArticlepeer-review

35 Scopus citations

Fingerprint

Dive into the research topics of 'Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene'. Together they form a unique fingerprint.

Keyphrases

Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science

Immunology and Microbiology

Neuroscience